ICER’s Final Evidence Report on bluebird bio’s Gene Therapy, beti-cel, Highlights Alternative Payment Model for One-Time Curative Treatment 

In July, the Institute for Clinical and Economic Review (ICER) published its final evidence report for bluebird bio’s Zynteglo (betibeglogene autotemcel or “beti-cel”) for the treatment of beta thalassemia, a rare, debilitating blood disorder.

The ICER assessment determined that beti-cel meets its value threshold for transfusion-dependent thalassemia (TDT) “at a cumulative price of $2.1 million if paid through an outcomes-based contract for patients with sustained transfusion independence.”

This demonstrates a continued focus on developing alternative payment and reimbursement models for innovative one-time therapies (like cell and gene therapies) to enable patient access. In order for beti-cel to meet ICER’s value threshold, the $2.1M price would need to come with a guarantee – an 80% payback for patients who do not achieve and maintain transfusion independence over five years (Phase III trials of beti-cel showed transfusion independence was achieved in 89% of the patients who received the gene therapy).

Why this matters:

  • While this is not the first ICER assessment on value and payment for gene therapies, it was published amid continued scrutiny of drug pricing, federal and state policy reforms and the FDA PDUFA review date for beti-cel scheduled for August 19.  
  • The Final Report provides bluebird bio with considerable momentum ahead of a final FDA decision, demonstrating, yet again, how ICER uses the timing of its review to influence key stakeholder decisions and launch pricing, as well as to influence payment and reimbursement decisions.  
  • Manufacturers of cell and gene therapies should anticipate that payers will demand arrangements based on outcomes performance for one-time therapies that often come with a high cost.
    • On the positive side, the assessment provides a path to manage payer uncertainties that come along with durable therapies – availability of long-term safety and efficacy data at approval – and enable patient access to transformative therapies in the US.
  • By contrast, bluebird bio pulled beti-cel from the European market in 2021 due to inability to secure adequate reimbursement (Germany offered $790,000, with the payout moving to $950,000 if the therapy was still working after five years; however, bluebird bio reportedly wanted something in the range of $1.8M).

What life science manufacturers can do: 

  • ICER continues to conduct reviews earlier in development to coincide with regulatory milestones to drive coverage and influence. Manufacturers should prepare proactive and reactive value communications accordingly. 
  • All companies nearing regulatory approval benefit from third-party validation of price, whether ICER agrees or disagrees with the price point. Working early with stakeholders to seed value narratives supports communications at approval.
  • Manufacturers with a vested interest in cell and gene therapies should pay careful attention to marketing communications and payer discussions. 

About the Author:

Patrick Rigby joined Syneos from the private sector where he specialized in corporate communication, government relations, and public affairs across the healthcare industry. Patrick served as Director of Communications and later as Chief of Staff for the New Jersey Office of Homeland Security and Preparedness under two administrations. He also served as an Advisor to the Chairman of the U.S. Senate Foreign Relations Committee. Prior to government service, Patrick held positions with Bloomberg L.P., the Council on Foreign Relations, U.S. House of Representatives, and in the financial services industry. Patrick brings with him over 15 years of managing large teams and directing complex communications and reputation management programs for businesses and government.